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The Future of BioPharma blog provides timely coverage of news that directly impacts the business strategies of the biotech, pharmaceutical and medical device industries. In addition to news coverage, the Future of BioPharma blog features live event coverage from IIR's Biopharmaceutical and Healthcare division.

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Monday, August 1, 2016

Researching and developing lifesaving medicines can take as long as 15 years and the costs are high – recently estimated at $2.6 billion by the Tufts Center for the Study of Drug Development. Venture capital can be hard to get, especially in the early stages of the drug pipeline. Broadview Ventures summed up the situation in their Venture Philanthropy in the Healthcare Marketplace report: “Venture capitalists are risk averse. NIH is retracting. Big Pharma is refocused on marketing and commercialization, as the productivity of R&D declines. This widening translational gap threatens to strand promising products and cures.”

Luckily venture philanthropy is increasingly fulfilling those financial needs with important bridge funding. And where once philanthropies focused on funding academic and institutional research, more and more are beginning to take a look at biotech companies, and essentially helping to build those bridges from bench to bedside in order to expedite the delivery and approval of life saving therapeutics. Chris Colecchi of Broadview Ventures was quoted in their report: “Venture philanthropy can disrupt the status quo with fewer dollars than people think. Modest amounts of money can fund early stage research in that translational space.” Indeed their research shows that 51% of the funding that venture philanthropies are giving focuses on the preclinical stage of development, a “key unmet need in the funding landscape”. But just exactly who is funding medical research, and what research are they funding? Here are examples of five Boston biotechs who are working with patient advocacy foundations to develop cures.

Five years ago the Juvenile Diabetes Research Foundation (JDRF) stepped in to help Boston biotech Selecta Biosciences develop a vaccine for type 1 diabetes, specifically a therapeutic which would prevent the autoimmune response that causes type 1 diabetes. Since JDRF’s inception, JDRF has contributed more than $2 billion to T1D research and is currently funding 50 human clinical trials of potential T1D therapies. Selecta’s ability to begin this research with the help of JDRF’s funding led them to the success they have now; just this June they raised $70 million in an IPO. Selecta describes their technology as being: “designed to communicate precise instructions to the immune system and to expand the use of vaccines to immunotherapies that enable a new generation of biologic therapies to treat autoimmune diseases, allergies, and cancer.” You can learn more about JDRF grants and the work they do here.

Akashi Therapeutics is unusual in that it was founded not by VCs but by nonprofit disease advocacy groups for the main purpose of developing a cure for Duchenne muscular dystrophy. Duchenne is a disease which affects about 300,000 people a year, primarily boys, and leads to progressive decline in muscle function and early death. Akashi Therapeutics was founded in 2010 – known then as Dart Therapeutics - and by 2014 it had raised $2.5 million dollars from 25 different patient advocacy groups. One of those groups was CureDuchenne. Akashi has had to suspend an experimental treatment called HT-100 for Duchenne following the tragic death of one child who was being given a high dose of the drug. (You can get an update from their CEO Marc Blaustein here where he talks about next steps for the HT-100 study.) Early this year CureDuchenne’s president and CEO Debra Miller spoke with Boston Business Journal about the situation: “We are confident that the team at Akashi will be able to isolate the problem soon. Anti-fibrotic drugs are needed, and CureDuchenne will continue to fund a research pipeline so that we can treat all aspects of this devastating disease.” CureDuchenne is currently funding 10 clinical trials and 7 preclinical studies as well as the Center for Duchenne Muscular Dystrophy at UCLA. You can apply for a research grant directly on their website here.

Early this year The Boston Globe described The Cystic Fibrosis Foundation and their then President Robert J Beall as having “pioneered” the model of “venture philanthropy”. In fact, The Cystic Fibrosis Foundation have invested a great deal of their resources in the Boston area: with early investment in Vertex Pharmaceuticals, a current alliance with Shire PLC and their own research lab in Bedford, Massachusetts. Late this spring Boston’s Editas Medicine made an exciting announcement – that The Cystic Fibrosis Foundation would be giving them up to $5 million for their research on CRISPR/Cas9 based medicines. CFF’s press release says that in 2015 they: ”gave approximately $15 million in grants to nearly 50 scientific laboratories to fund CF research on emerging technologies such as gene editing, gene delivery and stem cell research.” Learn more about CFF research awards here.

Last year Emulate was awarded a grant from the Michael J. Fox Foundation to apply the company’s “organs-on-chips” for the investigation of drug candidates for Parkinson’s disease. Organs on chips basically simulate the real thing for testing, to give more predictive data as well as a deeper understanding of both drug and disease. This funding led to Emulate eventually raising $28 million in a Series B Round this past March, as well as partnerships with both Merck and Johnson & Johnson. The Michael J. Fox Foundation website shows recent grants to Boston’s own MGH and MIT, and their press says they have funded more than $600 million in research to date fundamentally altering the “trajectory of progress toward a cure” for Parkinson’s disease. You can learn more about MJFF grants here.

Back in 2012, Constellation Pharmaceuticals began a partnership with The Leukemia & Lymphoma Society (LLS) to “advance the development of a novel BET inhibitor.” LLS provided $7.5 million in funding for their Phase I trial. Since then Constellation has established an alliance with Genentech and gone on to get $55 million in financing from venture capital. Their website describes why they develop drugs that target epigenetic function: “Our research has shown that when epigenetic regulatory events occur aberrantly, the proteins that regulate these events can become drivers of disease. Inhibiting these targets with novel agents promises to be a powerful avenue to develop important treatments serving unmet medical needs.” LLS invested a total of $67.2 million in cancer research last year. You can learn more about partnering with LLS here.

Please follow us on LinkedIn at our company page or on Twitter @BiotechWkBoston for more Boston biotech news and information, and don’t forget to check in every week for our Biotech Week Boston blog series. Biotech Week Boston is happening this October 4-7; you can learn more by clicking the link below.